Horizon Therapeutics announced the availability of an expanded access program for its investigational medicine teprotumumab.
Developed in partnership with the U.S. Food and Drug Administration (FDA), the expanded access program may provide access to teprotumumab for people living with an active thyroid eye disease (TED) who meet protocol criteria. The expanded access program will be available for a limited time while the FDA reviews Horizon’s Biologics License Application (BLA) for teprotumumab. Teprotumumab is an investigational medicine and its safety and efficacy have not been established.
According to the FDA, expanded access programs – sometimes called “compassionate use” – provide a pathway for a patient to receive an investigational medicine for a serious disease or condition. They are often made available when there are no comparable or satisfactory alternative therapies to treat the disease or condition; patient enrollment in clinical trials is not possible; potential patient benefit justifies the potential risk of treatment and providing the investigational medicine will not interfere with investigational trials that could support the medicine’s marketing approval for the treatment indication.